Figure 3.

Hexosaminidase Activity Is Restored in the CNS and Liver of Treated SD Mice

Mice (n = 6) from the high-dose cohorts were used (4 × 10¹² vg for AAV9 vectors and 1 × 10¹² vg for AAV-PHP.B vectors). Hexosaminidase activity (fold normal) in (A) CNS and (B) liver using artificial substrates 4-MUG and 4-MUGS hydrolyzed by all isozymes (total hexosaminidase) and HexA, respectively. (C) Isozyme analysis in liver of AAV treated SD mice and controls show peaks of 4-MUG activity in fractions 3, 15, and 22 corresponding to HexB (ββ), HexA (αβ), and HexS (αα), respectively. Treatment groups were compared to untreated SD mice using one-way ANOVA with Dunnett’s multiple comparison test for each tissue: ∗p < 0.05; ∗∗p < 0.01; ∗∗∗p < 0.001; ∗∗∗∗p < 0.0001.